IL282191A - שיטות ותכשירים לעריכת רנ" א - Google Patents

שיטות ותכשירים לעריכת רנ" א

Info

Publication number
IL282191A
IL282191A IL282191A IL28219121A IL282191A IL 282191 A IL282191 A IL 282191A IL 282191 A IL282191 A IL 282191A IL 28219121 A IL28219121 A IL 28219121A IL 282191 A IL282191 A IL 282191A
Authority
IL
Israel
Prior art keywords
compositions
methods
rnas
editing
editing rnas
Prior art date
Application number
IL282191A
Other languages
English (en)
Original Assignee
Univ Beijing
Edigene Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=70163607&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=IL282191(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Univ Beijing, Edigene Inc filed Critical Univ Beijing
Publication of IL282191A publication Critical patent/IL282191A/he

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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06Antianaemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/04Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0634Cells from the blood or the immune system
    • C12N5/0636T lymphocytes
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2320/34Allele or polymorphism specific uses
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    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/15011Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2800/00Nucleic acids vectors
    • C12N2800/10Plasmid DNA
    • C12N2800/106Plasmid DNA for vertebrates
    • C12N2800/107Plasmid DNA for vertebrates for mammalian

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Virology (AREA)
  • Cardiology (AREA)
  • Hematology (AREA)
  • Cell Biology (AREA)
  • Diabetes (AREA)
  • Hospice & Palliative Care (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
IL282191A 2018-10-12 2021-04-08 שיטות ותכשירים לעריכת רנ" א IL282191A (he)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CN2018110105 2018-10-12
CN2019082713 2019-04-15
PCT/CN2019/110782 WO2020074001A1 (en) 2018-10-12 2019-10-12 Methods and Compositions for Editing RNAs

Publications (1)

Publication Number Publication Date
IL282191A true IL282191A (he) 2021-05-31

Family

ID=70163607

Family Applications (1)

Application Number Title Priority Date Filing Date
IL282191A IL282191A (he) 2018-10-12 2021-04-08 שיטות ותכשירים לעריכת רנ" א

Country Status (21)

Country Link
US (2) US20210355494A1 (he)
EP (3) EP3864152B1 (he)
JP (2) JP7252328B2 (he)
KR (1) KR102666695B1 (he)
CN (4) CN113631708B (he)
AU (2) AU2019357450B2 (he)
BR (1) BR112021006844A8 (he)
CA (1) CA3115864A1 (he)
CL (1) CL2021000880A1 (he)
CO (1) CO2021005956A2 (he)
CR (1) CR20210243A (he)
DK (1) DK3864152T5 (he)
EC (1) ECSP21033637A (he)
ES (1) ES2962434T3 (he)
IL (1) IL282191A (he)
MX (1) MX2021004187A (he)
PE (1) PE20211402A1 (he)
PH (1) PH12021550805A1 (he)
SG (1) SG11202103666PA (he)
TW (1) TW202028466A (he)
WO (1) WO2020074001A1 (he)

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CN118416088A (zh) 2017-03-03 2024-08-02 加利福尼亚大学董事会 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
PE20211402A1 (es) * 2018-10-12 2021-08-02 Univ Beijing Metodos y composiciones para editar acidos ribonucleicos
JP2022518731A (ja) 2019-01-22 2022-03-16 コロ バイオ, インコーポレイテッド Rna編集オリゴヌクレオチド及びその使用
AU2020210900B2 (en) 2019-01-22 2025-04-24 Korro Bio, Inc. RNA-editing oligonucleotides and uses thereof
US11453878B2 (en) 2019-01-22 2022-09-27 Korro Bio, Inc. RNA-editing oligonucleotides and uses thereof
AU2020259548B2 (en) 2019-04-15 2023-10-12 Edigene Therapeutics (Beijing) Inc. Methods and compositions for editing RNAs
EP3997229A4 (en) 2019-07-12 2024-07-03 Peking University TARGETED RNA EDITING BY HARNESSING ENDOGENOUS ADAR USING MODIFIED RNA
JP2023504314A (ja) 2019-12-02 2023-02-02 シェイプ セラピューティクス インコーポレイテッド 治療的編集
PH12022551594A1 (en) 2019-12-30 2023-11-29 Edigene Therapeutics Beijing Inc Method for treating usher syndrome and composition thereof
WO2021136408A1 (zh) 2019-12-30 2021-07-08 博雅辑因(北京)生物科技有限公司 一种基于leaper技术治疗mps ih的方法和组合物
TW202204621A (zh) * 2020-04-15 2022-02-01 大陸商博雅輯因(北京)生物科技有限公司 治療赫勒氏綜合症的方法和藥物
CA3173012A1 (en) * 2020-04-22 2021-10-28 Susan BYRNE Compositions and methods using snrna components
KR20230033651A (ko) * 2020-05-28 2023-03-08 코로 바이오, 인크. Serpina1의 adar-매개 편집을 위한 방법 및 조성물
CN113897359A (zh) * 2020-07-06 2022-01-07 博雅辑因(北京)生物科技有限公司 一种改善的rna编辑方法
WO2022007803A1 (zh) 2020-07-06 2022-01-13 博雅辑因(北京)生物科技有限公司 一种改善的rna编辑方法
IL278401A (he) 2020-10-29 2022-05-01 Yeda Res & Dev פולינוקלאוטידים לעריכה של רנ" א ושיטה לשימוש בהם
EP4240338A4 (en) * 2021-01-04 2025-08-06 Univ California IN VIVO PROGRAMMABLE RNA EDITING BY RECRUITMENT OF ENDOGENOUS ADARS
WO2022150974A1 (en) * 2021-01-12 2022-07-21 Peking University Targeted rna editing by leveraging endogenous adar using engineered rnas
AU2022291759A1 (en) * 2021-06-15 2023-12-21 Massachusetts Institute Of Technology Deaminase-based rna sensors
WO2023278436A1 (en) * 2021-06-29 2023-01-05 Shape Therapeutics Inc. Engineered guide rnas and polynucleotides
CN118202045A (zh) * 2021-08-18 2024-06-14 北京大学 工程化的adar募集rna及其使用方法
WO2023060186A1 (en) 2021-10-07 2023-04-13 Hepatx Corporation Methods of tracking donor cells in a recipient
KR20240134108A (ko) * 2021-10-29 2024-09-06 듀크 유니버시티 Rna-프로그램가능한 세포 편집을 위한 조성물 및 시스템 및 이의 제조 및 사용 방법
WO2023143539A1 (en) * 2022-01-28 2023-08-03 Edigene Therapeutics (Beijing) Inc. Engineered adar-recruiting rnas and methods of use thereof
WO2023185231A1 (en) * 2022-04-02 2023-10-05 Edigene Therapeutics (Beijing) Inc. Engineered adar-recruiting rnas and methods of use for usher syndrome
WO2024228735A2 (en) * 2022-07-15 2024-11-07 Georgia Tech Research Corporation Anti-sars-cov-1 and anti-sars-cov-2 activatable rnase guide sequences
KR20250092211A (ko) * 2022-10-18 2025-06-23 리제너론 파마슈티칼스 인코포레이티드 Rna 편집을 위한 프로그래밍 가능한 snrna를 포함하는 조성물 및 방법
CN118620958A (zh) * 2023-03-08 2024-09-10 广州医科大学 一种抗原表位的制备方法
WO2024197951A1 (zh) * 2023-03-31 2024-10-03 时夕(广州)生物科技有限公司 一种与rna编辑靶位点形成编辑底物的序列
CN118853661A (zh) * 2023-04-28 2024-10-29 时夕(广州)生物科技有限公司 编辑rna的方法、组合物及其应用
WO2024235283A1 (en) * 2023-05-16 2024-11-21 Peking University Method of modulating rna splicing
CN119505015B (zh) * 2024-11-15 2025-10-17 北京大学 一种融合蛋白及其应用方法

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KR101297830B1 (ko) * 2010-09-10 2013-08-19 한국생명공학연구원 신규한 rna 결합 단백질 및 이를 이용한 무표지 마이크로 rna 검출방법
CN107532162A (zh) 2014-12-12 2018-01-02 托德·M·伍尔夫 用于利用寡核苷酸编辑细胞中核酸的组合物和方法
CN107109413B (zh) * 2014-12-17 2021-03-09 ProQR治疗上市公司Ⅱ 靶向的rna编辑
WO2017010556A1 (ja) 2015-07-14 2017-01-19 学校法人福岡大学 部位特異的rna変異導入方法およびそれに使用する標的編集ガイドrnaならびに標的rna-標的編集ガイドrna複合体
WO2017040477A1 (en) * 2015-08-31 2017-03-09 The University Of Chicago Composition and methods for detecting adenosine modifications
DE102015012522B3 (de) * 2015-09-26 2016-06-02 Eberhard Karls Universität Tübingen Verfahren und Substanzen zur gerichteten RNA-Editierung
KR102418185B1 (ko) * 2016-06-22 2022-07-06 프로큐알 테라퓨틱스 Ⅱ 비.브이. 단일 가닥 rna-편집 올리고뉴클레오타이드
WO2018041973A1 (en) * 2016-09-01 2018-03-08 Proqr Therapeutics Ii B.V. Chemically modified single-stranded rna-editing oligonucleotides
EP3571300A1 (en) 2017-01-19 2019-11-27 ProQR Therapeutics II B.V. Oligonucleotide complexes for use in rna editing
CN118416088A (zh) 2017-03-03 2024-08-02 加利福尼亚大学董事会 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
AU2018234825B2 (en) * 2017-03-15 2020-12-17 Massachusetts Institute Of Technology Novel CAS13B orthologues CRISPR enzymes and systems
AU2018338318B2 (en) * 2017-09-21 2022-12-22 Massachusetts Institute Of Technology Systems, methods, and compositions for targeted nucleic acid editing
PE20211402A1 (es) * 2018-10-12 2021-08-02 Univ Beijing Metodos y composiciones para editar acidos ribonucleicos

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EP3864152B1 (en) 2023-08-16
ECSP21033637A (es) 2021-08-31
TW202028466A (zh) 2020-08-01
US20210355494A1 (en) 2021-11-18
AU2023202184B2 (en) 2025-06-12
SG11202103666PA (en) 2021-05-28
PE20211402A1 (es) 2021-08-02
BR112021006844A8 (pt) 2023-03-21
AU2019357450A1 (en) 2021-06-03
CO2021005956A2 (es) 2021-07-30
KR102666695B1 (ko) 2024-05-23
PH12021550805A1 (en) 2021-10-04
AU2023202184A1 (en) 2023-05-04
US20210310026A1 (en) 2021-10-07
EP4306116A3 (en) 2024-04-17
WO2020074001A1 (en) 2020-04-16
CR20210243A (es) 2021-10-07
ES2962434T3 (es) 2024-03-19
CN115651927B (zh) 2023-06-16
MX2021004187A (es) 2021-09-08
KR20210076082A (ko) 2021-06-23
AU2019357450B2 (en) 2023-01-12
JP2021534810A (ja) 2021-12-16
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